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Latest News: Research

Cytokinetics Drug Awarded Orphan Drug Designation in the US

25 May 2017 / Posted in: Research

Cytokinetics has announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to its compound CK-2127107 for the potential treatment of SMA.

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Read the Latest Responses from Biogen about Nusinersen in the UK

16 May 2017 / Posted in: Research

Biogen responds to NHS England's requests about the UK EAP for infants with SMA Type 1; they also tell us when they hope to hear from NICE.

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Biogen Advises on Future Access Position for Children Enrolled in an SMA Type 1 EAP

11 May 2017 / Posted in: Research

We asked about the position in the UK and overseas.

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Update on NHS England's position re EAP for SMA Type 1

10 May 2017 / Posted in: Research

You can now read the latest information about NHS England’s position on considering the possibility of supporting the costs of delivering the EAP in England. We now have authority to share information from the meeting we attended on 25th April with the NHS England Specialised Commissioning team.

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Cytokinetics Initiates Second Cohort in Phase 2 Trial of CK-2127107

26 April 2017 / Posted in: Research

Cytokinetics has announced that it has initiated enrolment of a second cohort of SMA patients with SMA Types 2, 3 or 4 in their phase 2 clinical trial of CK-2127107, in North America.

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Update on CHERISH and NURTURE clinical trials of Spinraza

25 April 2017 / Posted in: Research

Biogen and Ionis Pharmaceutical have released information on the CHERISH and NURTURE clinical trials of their gene therapy drug, Spinraza (formerly nusinersen).

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EMA recommends marketing authorisation for nusinersen

21 April 2017 / Posted in: Research

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the marketing approval of nusinersen.

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New Clinical Care Research Article: Scoliosis in SMA

18 April 2017 / Posted in: Research

This new research article looks at recent research into different aspects of spinal surgery, in the treatment of scoliosis in Spinal Muscular Atrophy.

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Biogen Announces EAP for Nusinersen Will Not Extend More Widely Than SMA Type 1

13 April 2017 / Posted in: Research

Biogen have set out their reasoning for this decision. We understand many families will be bitterly disappointed by this news.

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Clinicians' Open Letter about the EAP for Children with SMA Type 1 in the UK

06 April 2017 / Posted in: Research

Today, seventy clinicians, physiotherapists, study coordinators, industry and advocacy groups' representatives, including SMA Support UK, outline their position and recommendations to NICE, NHS England's Care and Clinical Reference Groups (CRGs), NHS Trusts and Biogen.

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